- To compare whether palifermin versus placebo administered to pediatric patients three
days prior to conditioning and three days after autologous or allogeneic hematopoietic
stem cell transplantation (HSCT) is associated with a reduction in the incidence of WHO
grade 3 or 4 oral mucositis.

Secondary

- To evaluate the safety and tolerability of palifermin.

- To evaluate the long-term effects of palifermin on disease outcome and survival.

- To compare the incidence, total dose, and duration of parenteral opioid analgesic use
(morphine equivalents), and incidence and duration of total parenteral nutrition (TPN)
administration in patients treated with these regimens.

- To compare the incidence of febrile neutropenia and invasive bacterial infections in
patients treated with these regimens.

- Arm I: Patients receive palifermin IV once daily for 3 days prior to chemotherapy
and/or radiotherapy in the absence of unacceptable toxicity. Patients then receive
palifermin IV on days 0, 1, and 2 after autologous or allogeneic HSCT.

- Arm II: Patients receive placebo IV once daily for 3 days prior to chemotherapy and/or
radiotherapy in the absence of unacceptable toxicity. Patients then receive placebo IV
on days 0, 1, and 2 after autologous or allogeneic HSCT.

Blood samples are collected at baseline, 32 days, and 100 days after HSCT to evaluate the
immunogenicity of palifermin. Oral mucositis is assessed at baseline, daily for 8 days prior
to and 32 days after HSCT, or until oral mucositis has resolved by the WHO Mucositis Scale,
Oral Mucositis Assessment Scale (OMAS), modified Walsh mucositis scale, Oral Mucositis Daily
Questionnaire (OMDQ), and the pain categorical rating scale.

After completion of HSCT, patients are followed periodically for up to 10 years.

Study Design:

Outcome Measure:

Incidence of WHO grade 3 or 4 oral mucositis

Outcome Description:

The incidence of WHO grade 3 or 4 mucositis, the palifermin and placebo groups, will be compared using a generalized Cochran-Mantel-Haenszel method for general association as the primary analysis. In addition, this outcome will be examined using a logistic regression model; both approaches will account for the randomization strata. Potential confounders will be examined using multiple logistic regression models.

Outcome Time Frame:

Up to day 32

Safety Issue:

Yes

Principal Investigator

Lillian Sung, MD, PhD

Investigator Role:

Study Chair

Investigator Affiliation:

The Hospital for Sick Children

Authority:

Unspecified

Study ID:

ACCL0521

NCT ID:

NCT00728585

Start Date:

January 2009

Completion Date:

January 2009

Related Keywords:

Breast Cancer

Graft Versus Host Disease

Kidney Cancer

Leukemia

Lymphoma

Mucositis

Multiple Myeloma

Plasma Cell Neoplasm

Myelodysplastic Syndromes

Neuroblastoma

Ovarian Cancer

Sarcoma

Testicular Germ Cell Tumor

mucositis

graft versus host disease

recurrent childhood large cell lymphoma

recurrent childhood lymphoblastic lymphoma

recurrent childhood small noncleaved cell lymphoma

recurrent/refractory childhood Hodgkin lymphoma

accelerated phase chronic myelogenous leukemia

blastic phase chronic myelogenous leukemia

childhood acute lymphoblastic leukemia in remission

childhood acute myeloid leukemia in remission

childhood chronic myelogenous leukemia

chronic phase chronic myelogenous leukemia

juvenile myelomonocytic leukemia

recurrent childhood acute lymphoblastic leukemia

recurrent childhood acute myeloid leukemia

secondary acute myeloid leukemia

childhood myelodysplastic syndromes

de novo myelodysplastic syndromes

disseminated neuroblastoma

previously treated childhood rhabdomyosarcoma

previously treated myelodysplastic syndromes

recurrent Wilms tumor and other childhood kidney tumors

recurrent childhood rhabdomyosarcoma

recurrent neuroblastoma

recurrent malignant testicular germ cell tumor

secondary myelodysplastic syndromes

stage I multiple myeloma

stage II multiple myeloma

stage III multiple myeloma

refractory multiple myeloma

stage I ovarian epithelial cancer

stage II ovarian epithelial cancer

stage III ovarian epithelial cancer

stage IV ovarian epithelial cancer

recurrent ovarian epithelial cancer

recurrent ovarian germ cell tumor

stage IIIA breast cancer

stage IIIB breast cancer

stage IIIC breast cancer

stage IV breast cancer

recurrent breast cancer

Breast Neoplasms

Neoplasms

Carcinoma, Renal Cell

Kidney Neoplasms

Graft vs Host Disease

Leukemia

Lymphoma

Multiple Myeloma

Neoplasms, Plasma Cell

Plasmacytoma

Myelodysplastic Syndromes

Preleukemia

Neuroblastoma

Ovarian Neoplasms

Stomatitis

Neoplasms, Germ Cell and Embryonal

Mucositis

Sarcoma

Name

Location

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